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Bloomsbury Genetic Therapies launched with £5m seed funding to develop potentially curative gene therapy treatments for rare neurological and metabolic diseases

London, United Kingdom, October 7, 2022 – Bloomsbury Genetic Therapies Limited (“Bloomsbury”), a biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases, based on clinically proven gene therapy technologies, today announced its launch with £5m seed funding led by UCL Technology Fund.

Bloomsbury’s innovative approach is designed to optimize therapeutic efficacy and safety, enable high manufacturability, accelerate development timelines and maximize regulatory success to create a pipeline of highly differentiated first-to-market or first-in-class programs. The Company’s main programs are gene therapies targeted at the liver and the CNS:

Liver-targeted therapies: BGT-OTCD: Ornithine transcarbamylase deficiency (OTCD)

CNS Targeted Therapies: BGT-DTDS: Dopamine Transporter Deficiency Syndrome (DTDS)

BGT-NPC: Niemann-Pick disease type C (NPC)

BGT-INAD: Infant Neuroaxonal Dystrophy (INAD)

These programs are underpinned by the unique medical knowledge derived from the internationally renowned clinics led by the company’s academic founders, a deep understanding of disease biology, and world-class translational science resulting from the close collaboration between the academic founders of Bloomsbury. In addition, the viral vectors of the Company’s programs have been designed with well-characterized adeno-associated virus (AAV) capsids that have been proven in clinical and/or commercial settings. They have also been combined with innovative cassettes and routes of administration designed for optimal therapeutic effect.

“Bloomsbury is developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases”, said Adrien Lemoine, co-founder and managing director of Bloomsbury. “Our approach was designed with one goal in mind – to deliver the promise of gene therapy to rare disease patients by leveraging our industry’s existing cutting-edge technology and we have already attracted a very talented team of founders and experienced scientists. and company professionals to this effect, as we progress through the clinic with our first liver-targeted gene therapy program, BGT-OTCD for the treatment of ornithine transcarbamylase deficiency.

“Our commitment to Bloomsbury is a great example of our strategy to build truly innovative businesses anchored by exceptional science and experienced teams,” added Leigh Brody, investment manager of the UCL Technology Fund. “We are excited about the potential to develop best-in-class gene therapy products. »

Bloomsbury was founded as a spin-out from UCL and was backed by UCL Business, which helped drive the progress of these translational projects, supporting the team while managing the intellectual property and making this deal possible. Bloomsbury draws on the cutting-edge gene therapy and rare disease expertise of the company’s academic founders:

  • Professor Paul Gissen – Professor in pediatric metabolic diseases at UCL. He is also Honorary Consultant and Head of Pediatric Metabolic Disease Research at Great Ormond Street Hospital (GOSH), Head of Niemann Pick Type C and Batten Disease Specialist Clinics, NIHR Principal Investigator and UK Chief Investigator for a number of industry clinics. trials
  • Professor Manju Kurian – Professor of Neurogenetics at UCL and NIHR Research Professor. She is also Head of Molecular Neurosciences at UCL Great Ormond Street Institute of Child Health (GOS ICH), Honorary Consultant in Pediatric Neurology at GOSH, Acceleration of New Therapies Theme Leader at GOSH Biomedical Research Council and Head of a Specialist Service internationally famous. in the UK for genetic movement disorders in children
  • Professor Ahad Rahim – Professor of Translational Neurosciences at UCL, Head of the Pharmacology Research Department and Wellcome Chair in Pharmacology at UCL and co-founder of the GeneTxNeuro Viral Vector Core Facility at UCL
  • Professor Simon Waddington – Professor of gene therapy at UCL and president of the therapeutic innovation network in cell and gene therapy at UCL

“After years of tireless work to identify and develop potential treatments for OTCD, NPC, DTDS and INAD, we are delighted to have partnered with the Bloomsbury team and are confident that these programs are in good hands to progress through the development phase and towards regulatory approvals, with the ultimate goal of delivering curative therapies to patients,” added Professors Gissen, Kurian, Rahim and Waddington.

The senior management

Bloomsbury is led by co-founder and CEO Adrien Lemoine, who brings 20 years of pharmaceutical and biotechnology experience, including commercial, strategic and operational roles at AstraZeneca and GSK and joined the company after Orchard Therapeutics where he held the position of Chief Business Officer. In this role, he played a leading role in the company’s fundraising efforts (Series B, Series C, NASDAQ IPO and follow-on financing) which raised over $750 million and built the pipeline of the company as well as setting up Orchard’s discovery research efforts, alongside managing the leadership and program management function.

Alongside him on the management team are:

  • John Ilette, who serves as chief operating officer and chief legal officer of Bloomsbury. John, who was most recently General Counsel and Chief of Staff at Orchard Therapeutics, has over 30 years of pharmaceutical and biotechnology experience, both in private practice and in-house, with biopharmaceutical companies such as Oxford GlycoSciences , Cephalon, BioMarin and Consort Medical. . His responsibilities have included building and leading legal, intellectual property and corporate compliance functions as well as managing human resources, quality, safety and facilities functions. John’s experience includes private and public fundraising, IPOs and secondary listings, international mergers and acquisitions, complex commercial and patent litigation, rare disease product launches, and licensing and licensing of a number of gene therapy products.
  • Leigh Shaw, who serves as Bloomsbury’s Senior Vice President, Regulatory Affairs and Non-Clinical GLP Safety/Toxicology. Leigh has 30 years of industry experience in the development of gene therapies and therapeutic vaccines for a range of neurological and orphan indications. Most recently, he held positions at Nightstar Therapeutics, United Neuroscience and GenSight Biologics where he played a leading role in the successful transition of products from research to the clinic, the completion of Phase 2 negotiations with the US FDA and the EMA, and the application for marketing authorization in the EU.

board of directors

Bloomsbury has also brought together an impressive trio of industry leaders as independent directors to help management drive the company forward; to know:

  • Frank Armstrong, who acts as Chairman of the Board of Directors. Frank has held CEO positions at five biotechnology companies, both public and private, including Fulcrum Pharma, CuraGen, Bioaccelerate, Provensis and Phoqus. He has also held senior positions at Merck Serono, Bayer and Zeneca. Frank is currently Chairman of BioCaptiva and Faron Pharmaceuticals, is a Non-Executive Director of Newcells Biotech and ECO Animal Health Group plc, and a member of the Senior Advisory Board of Healthcare Royalty Partners.
  • Michael Diem, who serves as an independent non-executive director. Michael is Chief Commercial Officer and Chief Financial Officer at Century Therapeutics. Prior to joining Century, he held leadership positions at Amicus Therapeutics, Aevi Genomic Medicine, AstraZeneca and GSK. He currently sits on the board of Venatorx Pharmaceuticals.
  • Andrea Spezzi, who serves as an independent non-executive director. Andrea is the co-founder, president and CEO of discovery-stage start-up, Rejuvitas Inc. She co-founded and served as chief medical officer of Orchard Therapeutics, where she was responsible for leading the successful development of Libmeldy®, and has also held leadership positions at Takeda and GSK.